ALS Research Reauthorization with Transparent Safety Review

This VFA proposal would amend the Accelerating Access to Critical Therapies for ALS Act of 2021. It would extend the existing $100 million annual authorization of appropriations through fiscal year 2031 and prohibit new awards of the Act's ALS expanded-access research grants on or after September 30, 2031, rather than September 30, 2026. The annual amount is the same as the authorization in current law; actual spending would still require Congress to appropriate funds. This proposal creates no entitlement to an investigational drug and does not change the Food and Drug Administration's expanded-access approval requirements. For the ALS research grants, the Department of Health and Human Services would retain the existing eligibility and safeguards: an expanded-access request must be allowed to proceed before a grant application, the research must use data from patients who are not otherwise eligible for clinical trials, the grant must not impede clinical-trial enrollment, and the project must allow equitable access for minority and underserved populations. On an application to renew a grant, HHS would request from the manufacturer or sponsor and assess enrollment, safety, and any available efficacy data for the investigational drug. A combined phase 2/3 trial would qualify as a participating clinical trial only if enrollment begins within the period set by HHS in the grant terms. These renewal and trial-definition provisions follow H.R. 8205. A grant applicant would have to commit to promptly reporting new and serious adverse events and unexpected safety information relating to the phase 3 trial to the grant-making institution, in addition to existing federal investigational-drug safety-reporting requirements. As an additional VFA safeguard, HHS would publish annual, aggregate information on grant awards, renewal decisions, and the timeliness of required safety reports. The public report would exclude patient-identifying information and protected trade secrets; it would not replace reports to FDA or the grant-making institution. The existing FDA grant-and-contract program for ALS and other rare neurodegenerative diseases and the HHS public-private partnership would continue under their current statutory terms. FDA would publish an updated rare-neurodegenerative-disease action plan within 18 months after enactment, describing work over the following five years, and publish a report on its actions within five years after enactment, as H.R. 8205 proposes. As an additional VFA measure, FDA would publish a plain-language explanation of the lawful expanded-access process and its limits for patients and families. HHS would administer these duties through its existing grant and regulatory authorities, subject to annual appropriations and applicable privacy and research-safety law. The added public reporting duty would be reviewed by the House Committee on Energy and Commerce and the Senate Committee on Health, Education, Labor, and Pensions. The prohibition on new ALS expanded-access research grants would begin on September 30, 2031 unless Congress extends the date; the rest of the 2021 Act would not expire solely because that grant authority ends.